Two phase III trials of new drugs for eosinophilic oesophagitis (EoE) have been reported in The New England Journal of Medicine. The findings not only highlight new treatments for the condition, but also potentially shift the emphasis for future therapeutic targets and diagnostic criteria.

In the first trial (EoE KIDS), Mirna Chehade and colleagues assessed dupilumab, a human monoclonal antibody that blocks IL-4 and IL-13 pathways, for EoE in patients aged 1–11 years. “Dupilumab was approved in the US to treat children with EoE 12 years and older in May 2022. FDA approval was then extended to treat children 1 year and older weighing at least 15 kg in January 2024,” explains Chehade, who adds that these new results provide the basis of this recent FDA approval. In this phase III trial, paediatric patients with active EoE (n = 102; mean age 7.1 years; 76% boys, 24% girls) were randomly assigned 2:2:1:1 to receive weight-tiered higher-exposure dupilumab, lower-exposure dupilumab or placebo for 16 weeks (part A), followed by a 36-week extension period active treatment phase in which eligible patients in each dupilumab group continued the same regimen and those in the placebo groups were assigned to either lower-exposure or higher-exposure dupilumab (part B).

《新英格兰医学杂志》报道了两项治疗嗜酸性粒细胞性食管炎（EoE）新药的 III 期试验。这些发现不仅强调了这种疾病的新治疗方法，而且还可能改变未来治疗目标和诊断标准的重点。

在第一项试验 (EoE KIDS) 中，Mirna Chehade 及其同事评估了 dupilumab（一种阻断 IL-4 和 IL-13 通路的人单克隆抗体）对 1-11 岁患者 EoE 的影响。 “Dupilumab 于 2022 年 5 月在美国获批用于治疗 12 岁及以上的 EoE 儿童。FDA 的批准随后于 2024 年 1 月扩大到治疗 1 岁及以上体重至少 15 公斤的儿童，”Chehade 解释道，他补充说，这些新药物结果为 FDA 最近的批准奠定了基础。在这项 III 期试验中，患有活动性 EoE 的儿科患者（ n = 102；平均年龄 7.1 岁；76% 男孩，24% 女孩）被随机分配为 2:2:1:1 接受体重分级较高暴露量的 dupilumab、较低剂量的 dupilumab 治疗。 - 暴露 dupilumab 或安慰剂 16 周（A 部分），然后是 36 周的延长期积极治疗阶段，其中每个 dupilumab 组中符合条件的患者继续相同的治疗方案，而安慰剂组中的患者被分配到较低暴露或较高暴露度的 dupilumab（B 部分）。